DNDi and Sanofi will donate the medicine in support of international efforts to eliminate the disease

Across many parts of Africa, Human African trypanosomiasis – commonly known as sleeping sickness – is a constant threat, particularly to those living in extreme poverty. Some 65 million people are at risk of contracting sleeping sickness from a parasite transmitted through the bite of a tsetse fly. Without prompt diagnosis and treatment, the parasite will invade the central nervous system and usually prove fatal.

Sleeping sickness has been targeted for elimination as a public health problem by the World Health Organization (WHO) by 2020. A lot of progress has been made in recent years, with fewer than 1,500 cases reported in 2017, the lowest in the last 75 years. However, current treatment requires patients to be transferred to sleeping sickness specialized centers, usually far from patients’ homes, which contributes to the difficulty of treatment. There is need for a simplified treatment that will allow patients to stay close to their homes.

This is about to change, thanks to an innovative partnership between the not-for profit Drugs for Neglected Disease initiative (DNDi) and Sanofi. Together they have developed a new all-oral treatment for sleeping sickness known as fexinidazole, which in clinical trials showed effectiveness against both the early stage of the disease as well as the later stage when the parasite has entered the brain.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recently adopted a positive scientific opinion of the drug under Article 58, which is intended to streamline the availability of high-priority medicines in lower-income countries outside Europe. Sanofi will donate fexinidazole to the WHO in the frame of the long term WHO-Sanofi partnership for neglected tropical diseases: treatment could begin reaching patients in 2019.

“Fexinidazole is a therapeutic breakthrough for patients, and Sanofi is proud to donate this medicine to the World Health Organization (WHO) as part of our mission to support the elimination of sleeping sickness,” said Dr. Ameet Nathwani, Chief Medical Officer and Executive Vice President Medical Function, Sanofi.

Advancing Care and Reaching More Patients

An all-oral treatment for sleeping sickness is a major advance in disease management.

The disease takes a greater toll than the physical effects on a patient: people with the disease, and their families, often encounter societal stigma, and as patients become unable to work it can lead to even greater poverty. Having a treatment that can be administered without requiring systematic hospitalization, is essential to achieving the goal of eliminating the disease - fexinidazole eliminates that issue.

Treatment can be started by trained healthcare workers who deliver a pill to patients in their own communities. The treatment takes 10 days, and those who are well enough can go home with the remainder of the pills to administer the rest themselves.

“I’ve dedicated my life as a doctor to sleeping sickness. An all-oral treatment has been a dream of mine for decades,” said Dr. Victor Kandé, who as Neglected Tropical Diseases Expert Advisor, Ministry of Health of the Democratic Republic of Congo (DRC), was principal investigator of the trials. “Those affected are some of the most vulnerable and live in some of the remotest areas of the Congo, if not the world. They need a treatment that is safe, effective and simple.”

A Shelved Compound Comes Back to Life

For years, sleeping sickness was treated with an arsenic-based drug that was extremely painful as well as toxic, killing five percent of the patients who received it. In 2009, a newer combination therapy was introduced, one that was far more effective and safe but also difficult to ship, store and administer, requiring patients to be hospitalized.

In 2005, DNDi began re-examining the potential of a compound that had been shelved in the 1980s by a company that, after a series of mergers, ended up under Sanofi’s umbrella. In 2009, Sanofi and DNDi signed a partnership to develop, manufacture and distribute fexinidazole.

The project’s $62.5 million cost was supported by seven European countries, as well as by private donors including the Bill & Melinda Gates Foundation and Médecins Sans Frontières. This project culminated in the EMA positive opinion that opens the door to treatment.

“Fexinidazole is an entirely new chemical entity that has been developed through an alternative non-profit R&D model. It is the first new chemical entity to be developed by DNDi,” said Dr. Bernard Pécoul, DNDi Executive Director. “This therapeutic breakthrough is testament to the unique partnership between DNDi and Sanofi to discover, develop and register a treatment for a severely neglected disease.”